Orphan Drugs and Rare Diseases

15th October to 16th October 2019,
London, United Kingdom

The market for rare disease products is continuously growing, expecting to reach $176 billion by 2020, with a CGR of 10.5% just for orphan drugs – this is twice the growth rate of the overall prescription drug market (5.3% CGR from 2014-2020).

Given this information, SMi are proud to announce our 9th Annual Orphan Drugs and Rare Diseases conference, aiming to bring together solution providers, biotechnology companies, clinical researchers, regulatory professionals and charity leaders together to discuss possible ways to accelerative orphan drug development and access to rare disease patients, including the introduction of recent technologies and products to help aid the access of orphan drugs.

Conference
programme

8:30 Registration & Coffee

9:00 Chairman's Opening Remarks

Dr Rick Thompson joined Findacure in 2015, after completing his PhD in Evolutionary Biology at the University of Cambridge. He began working as Findacure’s first Scientific Officer, helping to develop the charity’s proof of concept study for the innovative drug repurposing social impact bond. Having been promoted to CEO in May 2017, Rick now oversees the charity’s strategy and development, oversees major funding applications, and manages the team, while still maintaining Findacure’s scientific projects. Rick is always keen to speak about Findacure’s work, and aims to encourage industry engagement with rare disease patient groups, promoting an open and collaborative approach to rare disease research and patient support.

9:10 Early access for orphan drugs

For many years, Tom has partnered with Pharma and Biotech companies to design their strategy for pre-approval access and develop global programs, allowing patients to gain access to often lifesaving treatments. Within this time, Tom has been involved in setting up and running over 200 global Early Access Programs.

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS. She has extensive experience in understanding the issues that face companies and other stakeholders when trying to secure access for medicines for the orphan and ultra-orphan conditions. These include developing innovative access arrangements in liaison with industry, clinicians, patients and the NHS.
Sheela has co-authored several papers that discuss HTA methods for assessing value of orphan medicines and presented at many conference issue panels on the subject.
Prior to joining NICE, she commissioned rare and ultra-orphan disease services in the NHS delivering improvements through collaborating with industry, clinicians and patient groups. During that time, she successfully decommissioned failing services and seamlessly transferred the care of patients to better quality services.
Sheela has a passion for partnership working and believes that collaboration across the sector is the key to delivering high quality outcomes for all.

• Discussing the success of NICE’s cost effective approach in putting new orphan drugs on the market
• Overview of new appraisal techniques, including HST (Highly Specialised Technology),
• Challenges faced by orphan drug product manufacturers
• Introduction of the budget impact threshold developed for assessment of ultra-orphan drugs

10:30 Morning Coffee

11:00 Collaborations in rare disease research and commercialisation

• Discussing how rare disease research and commercialization differs due to the variety of cases
• More interlinks needed between patients, clinicians, scientists, authorities and industry professionals both nationally and internationally
• Addressing importance of close collaborations with patients when mapping out the patient’s journey
• Highlighting benefits of understanding rare disease
• Intervention of pharmaceutical companies to develop appropriate lifelong treatments

Katrin Jack, Strategic Planning Lead, Takeda Pharma AG

11:40 Drug repurposing – a lifeline for rare disease patients

Dr Rick Thompson joined Findacure in 2015, after completing his PhD in Evolutionary Biology at the University of Cambridge. He began working as Findacure’s first Scientific Officer, helping to develop the charity’s proof of concept study for the innovative drug repurposing social impact bond. Having been promoted to CEO in May 2017, Rick now oversees the charity’s strategy and development, oversees major funding applications, and manages the team, while still maintaining Findacure’s scientific projects. Rick is always keen to speak about Findacure’s work, and aims to encourage industry engagement with rare disease patient groups, promoting an open and collaborative approach to rare disease research and patient support.

• Exploring the commercial potential for drug repurposing and successes of rare disease patient groups in helping to drive these projects to the clinic
• Current developments in the field of social investment to fund generic drug repurposing and its potential for ultra-rare diseases
• Discussing how to encourage a research interest in rare and ultrarare diseases, potentially leading to the development of new treatments

12:20 Networking Lunch

13:20 Building an educational digital community in rare diseases - a PAC first Mentality

14:00 A collaborative model to improve the success of translational research: a charity’s perspective

Annamaria started her career as Technology Transfer Officer at UNIMITT, the University Centre for Innovation and Technological Transfer of the University of Milan. Since 2009 she has been working at Fondazione Telethon, first as Intellectual Property Manager. She is currently Head of Technology Transfer and coordinates IP management, marketing and out-licensing activities in the context of the Telethon Institute of Genetics and Medicine (TIGEM), the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and other initiatives partnered by Fondazione Telethon, including the Sofinnova Telethon Fund, the first Italian biotech investment fund recently launched by by Sofinnova Partners and Fondazione Telethon.

• Enabling factors and challenges in the development of an advanced therapy medicinal product
• How a charity is assisting families participating in gene therapies
• Can we catalyse the development” in series” of multiple therapies for genetic diseases?

Krzysztof Potempa is Founder and CEO at BRAINCURES. He is a passionate computational neuroscientist with over 10 years of experience in deciphering biology from big data in academia and industry. In August 2016, he established BRAINCURES, to enable biology-driven patient-treatment matching (i.e., precision medicine) based on targets, compounds and biomarkers that are associated with their novel molecular framework for brain function. Their unique biological intelligence approach and Discovery Engine Platform processes to de-risk and accelerate drug discovery, pipeline management and clinical decision making have been recognized with the Beanstalks 2017 Best Mental Health Startup- and Sachs BEF18 Rising Stars- Award.

• Overview of Braincure’s discovery engine – a unique and biology-powered in silico approach to accelerate preclinical drug discovery
• Case study on the molecular framework of BDE, how it does not require data-training associated with AI
• Pipeline of translational opportunities for digital technology and low cost/high success precision medicine

15:50 Registries, Apps, Wearables - How Does Real World Data Change the Way We Do Studies?

Elin Haf Davies PhD began her career as a Children’s Nurse, before going on to gain her PhD at University College London. She was part of the paediatric team at the European Medicine Agency responsible for implementing the Paediatric Regulation in Europe. After 6 years as a regulator Elin left to start aparito, a digital health company providing remote patient monitoring specifically for clinical trials. Elin is also part of the IMI consortium ADAPTSMART, focusing on adaptive pathways.

• Regulatory framework
• Technology tools
• Case studies

16:30 Chairman’s Closing Remarks and Close of Day One

Dr Rick Thompson joined Findacure in 2015, after completing his PhD in Evolutionary Biology at the University of Cambridge. He began working as Findacure’s first Scientific Officer, helping to develop the charity’s proof of concept study for the innovative drug repurposing social impact bond. Having been promoted to CEO in May 2017, Rick now oversees the charity’s strategy and development, oversees major funding applications, and manages the team, while still maintaining Findacure’s scientific projects. Rick is always keen to speak about Findacure’s work, and aims to encourage industry engagement with rare disease patient groups, promoting an open and collaborative approach to rare disease research and patient support.

8:30 Registration & Coffee

9:00 Chairman's Opening Remarks

At Alexion, Mike Page leads a global team which develops and executes of regulatory strategies across the company’s rare disease product portfolio. Prior to joining Alexion, Mike led the US Regulatory team for oncology at Eisai and the regulatory aspects of Eisai’s monoclonal antibody products. Prior to that, Mike was a regulatory affairs consultant at United BioSource Corporation, beforew which he was a Director of Regulatory Strategy at Pfizer, both in the UK and the US. Focusing mainly on late stage development and product registration, Mike has global experience in therapeutic areas including oncology, hematology, psychiatry, neurology, sexual health and addiction disorders.

9:10 Overview of current orphan drug regulation

At Alexion, Mike Page leads a global team which develops and executes of regulatory strategies across the company’s rare disease product portfolio. Prior to joining Alexion, Mike led the US Regulatory team for oncology at Eisai and the regulatory aspects of Eisai’s monoclonal antibody products. Prior to that, Mike was a regulatory affairs consultant at United BioSource Corporation, beforew which he was a Director of Regulatory Strategy at Pfizer, both in the UK and the US. Focusing mainly on late stage development and product registration, Mike has global experience in therapeutic areas including oncology, hematology, psychiatry, neurology, sexual health and addiction disorders.

• Discussion on current state regulation of orphan drugs, and the level of scientific rigour needed for orphan drug approval (accepting small patient group studies and short studies)
• Assessing the importance of clinical evidence during the launch of an orphan drug, taking into account the difficultly in planning and executing clinical trials for rare/ultra-rare diseases.
• Synopsis of the evidence requirements before and after the registration and open access to a new drug

9:50 Improving patient access to orphan medicinal products

Jayne Spink is Chief Executive of Genetic Alliance UK and Chair of Rare Disease UK. Jayne has a BSc and PhD in genetics and postdoctoral experience in molecular genetics and cell biology. She left the research bench to join the Department of Health, initially as scientific secretary to the Gene Therapy Advisory Committee. As Head of Genetic Science, Safety and Regulation, she has worked on policy areas that have included gene therapy clinical research, stem cell research, genetic testing, genetics and insurance and ethics. Jayne is a former Associate Director of the Centre for Clinical Practice at NICE and has more than a decade of senior-level experience in the third sector. Immediately prior to joining Genetic Alliance UK, she served for 5 years as CEO of the Tuberous Sclerosis Association and served as the Multiple Sclerosis Society’s Director of Policy and Research (also for 5 years).

• Current developments of approaches to help educate orphan drug manufacturers and stake holders
• Highlighting the benefits of the collaboration of patient panels to provide a more targeted approach to orphan drug development.
• Exploring the influence of patient groups in providing early access of orphan treatments for rare disease cases where there’s no alternative
• Importance of investing in rare disease spaces to provide future opportunities

10:30 Morning Coffee

11:00 MoCA early dialogue between payers and manufacturers: An Opportunity to Improve Decision Making for Orphan Drugs in Europe?

Martina’s current research includes methods and applications of Multi-Criteria Decision Analysis (MCDA) approaches in healthcare decision making; economic issues posed by the development and provision of treatments for rare diseases; value based assessments and HTA systems in high and middle income countries. This expertise informs projects undertaken for global pharmaceutical and life sciences companies, non-profit research organizations, charities and trade associations.
Martina joined the Office of Health Economics in 2004, following a placement at NICE in London. She has an MSc in Economics from the University of York and a first degree in economics from the University of Cagliari in Italy.

• What is the MoCa process and its aim? Which parties are involved and what are the applications to date?
• What is the Transparent Value Framework, how is it used within MoCA, and how can it be improved?
• What are the advantages and disadvantages of participating in MoCa from different perspectives?
• Final reflections on pan-European assessment for orphan drugs

11:40 Technology and orphan drug development

• Experiences and perspectives on the various challenges in the development of orphan drugs & the available facilitating and accelerating toolbox
• Analysing the challenges associated with the data sharing and analysis for rare disease research
• Changes in market prospects for rare diseases following the improvement in efficiency of drug development

Maria Pascual, Chief Regulatory Officer, Minoryx Therapeutics S.L.

12:20 Networking Lunch

13:20 DevelopAKUre: a successful drug re-purposing programme sponsored by the European Union

Dr Nicolas Sireau is the CEO and Chair of Trustees at the AKU Society, a patient group that helps people with AKU (short for alkaptonuria), a rare genetic disease affecting both his children. He is also co-founder and Chair of Findacure, an organisation that helps rare disease patient groups. Previously, Nick was the CEO of SolarAid, an NGO working in Africa. He is a fellow of the Ashoka Fellowship of Social Entrepreneurs and has a PhD in the social psychology of social movements. He is the editor of ‘Rare Diseases: Challenges and Opportunities for Social Entrepreneurs’ (Greenleaf 2013) and co-editor of the 'Patient Group Handbook: A Practical Guide for Research and Drug Development' (Findacure 2016). He is also the founder and Chair of Orchard - Advancing Global Research into obsessive-compulsive disorder (OCD).

Dr Anthony Hall (Tony) graduated from King’s College London with first class honours in physiology and pharmacology before going on to qualify as a doctor in 1991 from the Royal Free. Tony has spent many years working on the development of drugs for rare diseases; during the past 5 years he has worked at Prosensa / Biomarin on the development of antisense oligonucleotides for the treatment of Duchenne muscular dystrophy and at Mereo BioPharma on the development of a monoclonal antibody for the treatment of osteogenesis imperfecta. He is currently Chief Medical Officer at Aparito.
Together with Findacure’s co-founder, he published a book entitled “The Patient Group Handbook: A Practical Guide for Research and Drug Development”. Tony is a Trustee of Findacure and sits on the Scientific Advisory Board of the rare diseases charity Duchenne UK.

14:00 Chiesi’s CASE STUDY: Holoclar

After a Bachelor degree in Molecular Biology, followed by a Master specialisation in Health Biology, I worked for the Scientific Forensic department of the Italian Police Force in Florence. Afterwards I worked for a Pharmaceutical company where I was responsible for both sales and marketing activities of their paediatric drugs portfolio. I then spent two years at the University of Amsterdam within the System Biology department where I carried research on cell cycle. In 2015 I joined Chiesi Farmaceutici as a Project Manager in the Rare Disease field and in 2017 I started my career as a Global Medical Advisor still in the rare Disease field specializing myself in the Advance Therapies Medicinal Products (ATMPs).

• Introduction of stem cell-based therapies into the rare disease space
• Case study – The first EMA stem cell based approved therapy for treating LSCD (Limbal stem cell deficiency): Holoclar, what has been done until now and where are we
• Peculiarity of the product: the Holoclar Medical Service

15:20 Afternoon Tea

15:50 Charting the path to diagnosis for patients with rare diseases

Senior International Manager with more than 19 years of leadership capabilities and in-depth pharmaceutical experience driving results in Medical Affairs, Marketing & Sales, Pricing and Reimbursement, Market Access at both global and local scale. Demonstrates ability to work effectively in matrix organizations and multicultural environments. Exposure to multiple therapeutic areas: Hematology, Inborn Errors of metabolism, Genetic diseases, Nephrology, Gastroenterology, Dermatology, Internal Medicine, Inflammation and Immunology, Cardiovascular, Rare Diseases, Metabolic Disorders, Primary and Secondary Immunodeficiencies, Hereditary angioedema.
Giacomo Baruchello, GM at Vertex Italy and former GM Shire SP Italy said about him: “Donatello has a remarkable background that includes science and business that makes him able to contribute on both fronts. A very committed person, his strong execution skills and mostly appreciated follow through on commitments attitude complete the profile of a promising future leader.”
Source: LinkedIn, public profile.

• Discussing the importance of partnership between pharmaceutical, digital enterprises and patient associations
• Present the Global Commission to end the diagnostic odyssey of children with Rare diseases project
• Discussing the importance of the adoption of disruptive technology and advancement of diagnostic solutions in rare disease space

16:30 Patient Panel Discussion: perspective on patient engagement

• What's the best way for organisations to make patients feel valued?
• How would you advise organisations on language and accessibility? Ie, people-first language, use of braille etc.
• How do you feel patient engagement in the UK is different and/or similar to the USA?
• Is your rare disease community proactive in pushing for the best treatment for your conditions?
• How long have you been involved in patient advocacy?
• Social media is massive these days - how can organisations best engage with patients online?

David Rose is an accomplished public speaker. He has an ultra-rare disease called Occipital Horn Syndrome. He discusses the ups and downs of living with an ultra-rare disease. He discusses all aspects of living with an ultra-rare disease, from studying to holidays. As well as his rare disease presentations, he also speaks on behalf of Great Ormond Street Hospital about his experiences using the hospital from a very young age up until his late teens.

Lizzie is a Medical Writer at NexGen Healthcare Communications and has over five years’ in experience in the pharmaceutical sector. Working with a number of large pharmaceutical clients, she has successfully delivered projects in a range of clinical indications.

Lizzie’s real passion in medical communications relates to patients and how best they can be supported to live alongside their condition(s). She regularly liaises with patients living with a rare disease in order to demystify clinical research that is published relating to its causes and treatments.

17:30 Chairman’s Closing Remarks and Close of Day Two

At Alexion, Mike Page leads a global team which develops and executes of regulatory strategies across the company’s rare disease product portfolio. Prior to joining Alexion, Mike led the US Regulatory team for oncology at Eisai and the regulatory aspects of Eisai’s monoclonal antibody products. Prior to that, Mike was a regulatory affairs consultant at United BioSource Corporation, beforew which he was a Director of Regulatory Strategy at Pfizer, both in the UK and the US. Focusing mainly on late stage development and product registration, Mike has global experience in therapeutic areas including oncology, hematology, psychiatry, neurology, sexual health and addiction disorders.

FEATURED SPEAKERS

Senior International Manager with more than 19 years of leadership capabilities and in-depth pharmaceutical experience driving results in Medical Affairs, Marketing & Sales, Pricing and Reimbursement, Market Access at both global and local scale. Demonstrates ability to work effectively in matrix organizations and multicultural environments. Exposure to multiple therapeutic areas: Hematology, Inborn Errors of metabolism, Genetic diseases, Nephrology, Gastroenterology, Dermatology, Internal Medicine, Inflammation and Immunology, Cardiovascular, Rare Diseases, Metabolic Disorders, Primary and Secondary Immunodeficiencies, Hereditary angioedema.
Giacomo Baruchello, GM at Vertex Italy and former GM Shire SP Italy said about him: “Donatello has a remarkable background that includes science and business that makes him able to contribute on both fronts. A very committed person, his strong execution skills and mostly appreciated follow through on commitments attitude complete the profile of a promising future leader.”
Source: LinkedIn, public profile.

Jayne Spink is Chief Executive of Genetic Alliance UK and Chair of Rare Disease UK. Jayne has a BSc and PhD in genetics and postdoctoral experience in molecular genetics and cell biology. She left the research bench to join the Department of Health, initially as scientific secretary to the Gene Therapy Advisory Committee. As Head of Genetic Science, Safety and Regulation, she has worked on policy areas that have included gene therapy clinical research, stem cell research, genetic testing, genetics and insurance and ethics. Jayne is a former Associate Director of the Centre for Clinical Practice at NICE and has more than a decade of senior-level experience in the third sector. Immediately prior to joining Genetic Alliance UK, she served for 5 years as CEO of the Tuberous Sclerosis Association and served as the Multiple Sclerosis Society’s Director of Policy and Research (also for 5 years).

Martina’s current research includes methods and applications of Multi-Criteria Decision Analysis (MCDA) approaches in healthcare decision making; economic issues posed by the development and provision of treatments for rare diseases; value based assessments and HTA systems in high and middle income countries. This expertise informs projects undertaken for global pharmaceutical and life sciences companies, non-profit research organizations, charities and trade associations.
Martina joined the Office of Health Economics in 2004, following a placement at NICE in London. She has an MSc in Economics from the University of York and a first degree in economics from the University of Cagliari in Italy.

At Alexion, Mike Page leads a global team which develops and executes of regulatory strategies across the company’s rare disease product portfolio. Prior to joining Alexion, Mike led the US Regulatory team for oncology at Eisai and the regulatory aspects of Eisai’s monoclonal antibody products. Prior to that, Mike was a regulatory affairs consultant at United BioSource Corporation, beforew which he was a Director of Regulatory Strategy at Pfizer, both in the UK and the US. Focusing mainly on late stage development and product registration, Mike has global experience in therapeutic areas including oncology, hematology, psychiatry, neurology, sexual health and addiction disorders.

Dr Rick Thompson joined Findacure in 2015, after completing his PhD in Evolutionary Biology at the University of Cambridge. He began working as Findacure’s first Scientific Officer, helping to develop the charity’s proof of concept study for the innovative drug repurposing social impact bond. Having been promoted to CEO in May 2017, Rick now oversees the charity’s strategy and development, oversees major funding applications, and manages the team, while still maintaining Findacure’s scientific projects. Rick is always keen to speak about Findacure’s work, and aims to encourage industry engagement with rare disease patient groups, promoting an open and collaborative approach to rare disease research and patient support.

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS. She has extensive experience in understanding the issues that face companies and other stakeholders when trying to secure access for medicines for the orphan and ultra-orphan conditions. These include developing innovative access arrangements in liaison with industry, clinicians, patients and the NHS.
Sheela has co-authored several papers that discuss HTA methods for assessing value of orphan medicines and presented at many conference issue panels on the subject.
Prior to joining NICE, she commissioned rare and ultra-orphan disease services in the NHS delivering improvements through collaborating with industry, clinicians and patient groups. During that time, she successfully decommissioned failing services and seamlessly transferred the care of patients to better quality services.
Sheela has a passion for partnership working and believes that collaboration across the sector is the key to delivering high quality outcomes for all.

Annamaria Merico

Head of Technology Transfer, Fondazione Telethon

Annamaria started her career as Technology Transfer Officer at UNIMITT, the University Centre for Innovation and Technological Transfer of the University of Milan. Since 2009 she has been working at Fondazione Telethon, first as Intellectual Property Manager. She is currently Head of Technology Transfer and coordinates IP management, marketing and out-licensing activities in the context of the Telethon Institute of Genetics and Medicine (TIGEM), the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) and other initiatives partnered by Fondazione Telethon, including the Sofinnova Telethon Fund, the first Italian biotech investment fund recently launched by by Sofinnova Partners and Fondazione Telethon.

Anthony Hall

Co-founder, Findacure Development

Dr Anthony Hall (Tony) graduated from King’s College London with first class honours in physiology and pharmacology before going on to qualify as a doctor in 1991 from the Royal Free. Tony has spent many years working on the development of drugs for rare diseases; during the past 5 years he has worked at Prosensa / Biomarin on the development of antisense oligonucleotides for the treatment of Duchenne muscular dystrophy and at Mereo BioPharma on the development of a monoclonal antibody for the treatment of osteogenesis imperfecta. He is currently Chief Medical Officer at Aparito.
Together with Findacure’s co-founder, he published a book entitled “The Patient Group Handbook: A Practical Guide for Research and Drug Development”. Tony is a Trustee of Findacure and sits on the Scientific Advisory Board of the rare diseases charity Duchenne UK.

Dan Donovan

CEO, RareLife Solutions

Dan Jeffries

Author and Patient Advocate, Me, Myself & Eye

David Rose

Business Development, Rare Revolution Magazine

David Rose is an accomplished public speaker. He has an ultra-rare disease called Occipital Horn Syndrome. He discusses the ups and downs of living with an ultra-rare disease. He discusses all aspects of living with an ultra-rare disease, from studying to holidays. As well as his rare disease presentations, he also speaks on behalf of Great Ormond Street Hospital about his experiences using the hospital from a very young age up until his late teens.

Donatello Crocetta

VP, Global Head of Rare Immunology Franchise, Takeda

Senior International Manager with more than 19 years of leadership capabilities and in-depth pharmaceutical experience driving results in Medical Affairs, Marketing & Sales, Pricing and Reimbursement, Market Access at both global and local scale. Demonstrates ability to work effectively in matrix organizations and multicultural environments. Exposure to multiple therapeutic areas: Hematology, Inborn Errors of metabolism, Genetic diseases, Nephrology, Gastroenterology, Dermatology, Internal Medicine, Inflammation and Immunology, Cardiovascular, Rare Diseases, Metabolic Disorders, Primary and Secondary Immunodeficiencies, Hereditary angioedema.
Giacomo Baruchello, GM at Vertex Italy and former GM Shire SP Italy said about him: “Donatello has a remarkable background that includes science and business that makes him able to contribute on both fronts. A very committed person, his strong execution skills and mostly appreciated follow through on commitments attitude complete the profile of a promising future leader.”
Source: LinkedIn, public profile.

Elin Haf Davies

Director & Founder, Empowering Children | Enabling Research

Elin Haf Davies PhD began her career as a Children’s Nurse, before going on to gain her PhD at University College London. She was part of the paediatric team at the European Medicine Agency responsible for implementing the Paediatric Regulation in Europe. After 6 years as a regulator Elin left to start aparito, a digital health company providing remote patient monitoring specifically for clinical trials. Elin is also part of the IMI consortium ADAPTSMART, focusing on adaptive pathways.

Jayne Spink

Chief Executive Officer , Genetic Alliance UK

Jayne Spink is Chief Executive of Genetic Alliance UK and Chair of Rare Disease UK. Jayne has a BSc and PhD in genetics and postdoctoral experience in molecular genetics and cell biology. She left the research bench to join the Department of Health, initially as scientific secretary to the Gene Therapy Advisory Committee. As Head of Genetic Science, Safety and Regulation, she has worked on policy areas that have included gene therapy clinical research, stem cell research, genetic testing, genetics and insurance and ethics. Jayne is a former Associate Director of the Centre for Clinical Practice at NICE and has more than a decade of senior-level experience in the third sector. Immediately prior to joining Genetic Alliance UK, she served for 5 years as CEO of the Tuberous Sclerosis Association and served as the Multiple Sclerosis Society’s Director of Policy and Research (also for 5 years).

Katrin Jack

Strategic Planning Lead, Takeda Pharma AG

Krzysztof Potempa

Founder and Director, BRAINCURES

Krzysztof Potempa is Founder and CEO at BRAINCURES. He is a passionate computational neuroscientist with over 10 years of experience in deciphering biology from big data in academia and industry. In August 2016, he established BRAINCURES, to enable biology-driven patient-treatment matching (i.e., precision medicine) based on targets, compounds and biomarkers that are associated with their novel molecular framework for brain function. Their unique biological intelligence approach and Discovery Engine Platform processes to de-risk and accelerate drug discovery, pipeline management and clinical decision making have been recognized with the Beanstalks 2017 Best Mental Health Startup- and Sachs BEF18 Rising Stars- Award.

Lizzie McFarlane

Medical Writer, NexGen Healthcare Communications

Lizzie is a Medical Writer at NexGen Healthcare Communications and has over five years’ in experience in the pharmaceutical sector. Working with a number of large pharmaceutical clients, she has successfully delivered projects in a range of clinical indications.

Lizzie’s real passion in medical communications relates to patients and how best they can be supported to live alongside their condition(s). She regularly liaises with patients living with a rare disease in order to demystify clinical research that is published relating to its causes and treatments.

Maria Pascual

Chief Regulatory Officer, Minoryx Therapeutics S.L.

Martina Garau

Associate Director, Office Of Health Economics

Martina’s current research includes methods and applications of Multi-Criteria Decision Analysis (MCDA) approaches in healthcare decision making; economic issues posed by the development and provision of treatments for rare diseases; value based assessments and HTA systems in high and middle income countries. This expertise informs projects undertaken for global pharmaceutical and life sciences companies, non-profit research organizations, charities and trade associations.
Martina joined the Office of Health Economics in 2004, following a placement at NICE in London. She has an MSc in Economics from the University of York and a first degree in economics from the University of Cagliari in Italy.

Michael Page

Executive Director, Portfolio Products, Alexion Pharmaceuticals Inc

At Alexion, Mike Page leads a global team which develops and executes of regulatory strategies across the company’s rare disease product portfolio. Prior to joining Alexion, Mike led the US Regulatory team for oncology at Eisai and the regulatory aspects of Eisai’s monoclonal antibody products. Prior to that, Mike was a regulatory affairs consultant at United BioSource Corporation, beforew which he was a Director of Regulatory Strategy at Pfizer, both in the UK and the US. Focusing mainly on late stage development and product registration, Mike has global experience in therapeutic areas including oncology, hematology, psychiatry, neurology, sexual health and addiction disorders.

Nick Sireau

Chair and CEO, AKU Society

Dr Nicolas Sireau is the CEO and Chair of Trustees at the AKU Society, a patient group that helps people with AKU (short for alkaptonuria), a rare genetic disease affecting both his children. He is also co-founder and Chair of Findacure, an organisation that helps rare disease patient groups. Previously, Nick was the CEO of SolarAid, an NGO working in Africa. He is a fellow of the Ashoka Fellowship of Social Entrepreneurs and has a PhD in the social psychology of social movements. He is the editor of ‘Rare Diseases: Challenges and Opportunities for Social Entrepreneurs’ (Greenleaf 2013) and co-editor of the 'Patient Group Handbook: A Practical Guide for Research and Drug Development' (Findacure 2016). He is also the founder and Chair of Orchard - Advancing Global Research into obsessive-compulsive disorder (OCD).

Philipp Gallwitz

Founding Partner, Admedicum Business for Patients

Rick Thompson

CEO, Findacure

Dr Rick Thompson joined Findacure in 2015, after completing his PhD in Evolutionary Biology at the University of Cambridge. He began working as Findacure’s first Scientific Officer, helping to develop the charity’s proof of concept study for the innovative drug repurposing social impact bond. Having been promoted to CEO in May 2017, Rick now oversees the charity’s strategy and development, oversees major funding applications, and manages the team, while still maintaining Findacure’s scientific projects. Rick is always keen to speak about Findacure’s work, and aims to encourage industry engagement with rare disease patient groups, promoting an open and collaborative approach to rare disease research and patient support.

Sara Carlot

Global Medical Advisor - Rare Disease, Chiesi Farmaceutici S. p. A.

After a Bachelor degree in Molecular Biology, followed by a Master specialisation in Health Biology, I worked for the Scientific Forensic department of the Italian Police Force in Florence. Afterwards I worked for a Pharmaceutical company where I was responsible for both sales and marketing activities of their paediatric drugs portfolio. I then spent two years at the University of Amsterdam within the System Biology department where I carried research on cell cycle. In 2015 I joined Chiesi Farmaceutici as a Project Manager in the Rare Disease field and in 2017 I started my career as a Global Medical Advisor still in the rare Disease field specializing myself in the Advance Therapies Medicinal Products (ATMPs).

Sheela Upadhyaya

Sheela Upadhyaya is currently the Associate Director of the Highly Specialised Technology program at NICE and is responsible for running the program to evaluate medicines and technologies for rare and ultra-rare conditions for commissioning in the NHS. She has extensive experience in understanding the issues that face companies and other stakeholders when trying to secure access for medicines for the orphan and ultra-orphan conditions. These include developing innovative access arrangements in liaison with industry, clinicians, patients and the NHS.
Sheela has co-authored several papers that discuss HTA methods for assessing value of orphan medicines and presented at many conference issue panels on the subject.
Prior to joining NICE, she commissioned rare and ultra-orphan disease services in the NHS delivering improvements through collaborating with industry, clinicians and patient groups. During that time, she successfully decommissioned failing services and seamlessly transferred the care of patients to better quality services.
Sheela has a passion for partnership working and believes that collaboration across the sector is the key to delivering high quality outcomes for all.

Tom Watson

Executive Vice President, Bionical Emas

For many years, Tom has partnered with Pharma and Biotech companies to design their strategy for pre-approval access and develop global programs, allowing patients to gain access to often lifesaving treatments. Within this time, Tom has been involved in setting up and running over 200 global Early Access Programs.

Victoria Abbott-Fleming

Founder and Chair, Burning Nights CRPS Support

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For membership enquiries: cdevaal@rarerevolutionmagazine.com
For sales and advertising: drose@rarerevolutionmagazine.com

Supporters

View detailsAmerican Pharmaceutical ReviewSupportershttp://www.americanpharmaceuticalreview.comStart receiving your free copy today. American Pharmaceutical Review is a review of the drug pipeline from the late discovery phase through manufacturing. American Pharmaceutical Review prides itself on having the most relevant, unbiased and informative editorial in the industry. You will find that all of our editorial is highly technical and written by government agencies, consultants, academics, and large pharmaceuticals companies. American Pharmaceutical Review covers several key topics that are important to drug production:Separations and purification, Drug Delivery, Biopharmaceutical Processing, Biopharmaceutical Development, Formulation development, Manufacturing, Microbiology, Instrumentation, Spectroscopy

View detailsBiocompareSupportershttp://www.biocompare.comBiocompare.com is the leading resource for up-to-date product information, product reviews, and new technologies for life scientists. Biocompare combines an in-depth knowledge of life science products and new technologies with the power of the Internet to offer scientists the most dynamic, relevant, and innovative media-based marketplace for life science information.

View detailsDrug Discovery TodaySupportershttp://www.drugdiscoverytoday.com/Drug Discovery Today covers the whole of the preclinical drug discovery process. The reviews are cutting edge, written by experts in their respective fields and cover all aspects of drug discovery from genomic and proteomic approaches, computational drug design, medicinal chemistry and the translation of these sciences to therapies.

View detailsLabiotech.euSupportershttps://goo.gl/DRE2GpLabiotech.eu is the leading digital media covering the European Biotech industry. Over 100,000 monthly visitors use it to keep an eye on the business and innovations in biotechnology. Hope you'll enjoy reading our stories!

View detailsPharmaceutical OutsourcingSupportershttp://www.pharmoutsourcing.comPharmaceutical Outsourcing, the journal dedicated to pharmaceutical and biopharmaceutical contract services. With regular sections on contract manufacturing, contract research, contract packaging, formulation/development services, contract analytical testing, APIs, stability testing, clinical research and other areas, we bring the most complete coverage of trends and issues in the industry to our 15,000 readers in North America.

View detailsPharmacircleSupportershttp://www.pharmacircle.comPharmaCircle is an innovative knowledge management company serving the current and future global leaders in the Pharmaceutical and Biotechnology related industries. PharmaCircle is a one stop information and analysis source for pipeline, products, clinical trials, drug delivery technologies, deals and acquisitions, company financials, venture capital investments, product sales, pharmaceutical services, news, patents and more….

View detailsPharmalicensingSupportershttp://www.pharmalicensing.comPharmalicensing (www.pharmalicensing.com) is the premier biopharmaceutical Open Innovation resource designed for professionals involved with partnering, licensing and business development worldwide.
Actively supporting all forms of partnering and in- and out-licensing activities, Pharmalicensing utilizes the unique online Showcase Profiling & Discover services, as well as its renowned Partnering Search service leveraging its global network of industry experts, to enable companies to identify and engage with appropriate partners around the world.
Pharmalicensing is actively utilized by many tens of thousands of industry professionals each month. Pharmalicensing is a division of Cognis Group, Inc

View detailspharmaphorumSupportershttp://www.pharmaphorum.compharmaphorum drives innovation within the pharmaceutical industry, by bringing healthcare together through a suite of media services that help produce and disseminate thought leadership, combined with an online platform for communicating messages to a global audience.Visit www.pharmaphorum.com

View detailsSelectScienceSupportershttp://www.selectscience.net/register?utm_source=Media-Partner&utm_medium=Website&utm_campaign=SMISelectScience is an independent, online information resource for the worldwide scientific community, and the home of trusted information for laboratory scientists. Discover the latest drug discovery and development technologies, products and techniques with product reviews, videos, application notes and news articles. Become a member for free today.

View detailsSwiss Biotech AssociationSupportershttp://www.swissbiotech.org/The Swiss Biotech Association (SBA) is the national industry association for biotechnology, including pharmaceuticals, diagnostics, agriculture, food, cosmetics, environmental biotechnology, and specialty chemicals. Members are companies active in modern biotechnology, such as R&D, Production, Marketing and Sales, Finance, Services and Consulting. SBA provides a networking platform for Life Science clusters, academic and federal institutions the like. Founded in March 1998, the Association grows steadily.

VENUE

Holiday Inn Kensington Forum

97 Cromwell Road , London, United Kingdom

Holiday Inn Kensington Forum is perfectly situated in one of London’s most luxurious and beautiful areas within South Kensington.

The hotel is just 2 minutes walk from Gloucester Road tube station for convenient travel to Hyde Park, London Eye, Tower Bridge plus more of London’s top attractions. There are also easy and direct links to some major transport hubs including Victoria, Kings Cross St Pancras, Paddington and Heathrow.

This distinctive hotel in south London has so much to offer to make all guests really feel at home. The latest Holiday Inn relaunch is not just about the new look and feel for the hotel but to offer guests more benefits during their stay including a pillow menu for extra comfort during their sleep and a curved shower rail for more spacious feel.

In addition to our 906 rooms, all business guests can take advantage of our meeting and conference facilities including High Speed Internet Access and unlimited Starbucks coffee at The Academy. Our hotel’s professional event planners are on board to help take the hard work and stress away from planning your next event.

So whether you in London on business or pleasure, make the Holiday Inn London Kensington Forum your first choice of hotel and book your accommodation for our lowest internet rate guarantee.

A number of our clients have been approached by third party organisations offering to book hotel rooms. We would advise that you do not book through them as they are not representing the SMi Group. SMi Group books all hotel rooms directly. If you are approached by a third party organisation then please contact us before making any bookings. If you have already booked a hotel room using a third party organisation, we would highly recommend contacting the hotel you were booked into to ensure a booking has been made for you. We would also advise you to please check the terms and conditions of the booking carefully.

2018 Orphan Drugs Conference Presentation by MHRA

2018 Orphan Drugs Conference Presentation by AstraZeneca

Media Partners

Rare Revolution Magazine

Giving RARE a voice
Rare Revolution Magazine is dedicated to providing exceptional articles of interest to the rare disease community. We provide a platform to the fantastic patient groups and charities that support over 7000 different rare conditions.
This free subscription digital magazine means international access to all.
You will find compelling voices from rare disease advocates and patients, articles from clinical, research and pharmaceutical teams and the latest in ‘RARE’ advancements.
Our community is growing both virtually and through our local networks and ecosystems.
Be part of the #rarerevolution.
To receive Rare Revolution Magazine in your inbox visit www.rarerevolutionmagazine.com to subscribe for free.
For content queries email: editor@rarerevolutionmagazine.com
For membership enquiries: cdevaal@rarerevolutionmagazine.com
For sales and advertising: drose@rarerevolutionmagazine.com

Gene Therapy Net

Gene Therapy Net is the information resource for basic and clinical research in gene therapy, and the site serves as a network in the exchange of gene therapy information and breaking news items. Visitors can keep track of the latest scientific papers, conference announcements, gene therapy jobs, regulations and guidelines

Media Partners

Biocompare

Biocompare.com is the leading resource for up-to-date product information, product reviews, and new technologies for life scientists. Biocompare combines an in-depth knowledge of life science products and new technologies with the power of the Internet to offer scientists the most dynamic, relevant, and innovative media-based marketplace for life science information.

Swiss Biotech Association

The Swiss Biotech Association (SBA) is the national industry association for biotechnology, including pharmaceuticals, diagnostics, agriculture, food, cosmetics, environmental biotechnology, and specialty chemicals. Members are companies active in modern biotechnology, such as R&D, Production, Marketing and Sales, Finance, Services and Consulting. SBA provides a networking platform for Life Science clusters, academic and federal institutions the like. Founded in March 1998, the Association grows steadily.

pharmaphorum

pharmaphorum drives innovation within the pharmaceutical industry, by bringing healthcare together through a suite of media services that help produce and disseminate thought leadership, combined with an online platform for communicating messages to a global audience.Visit www.pharmaphorum.com

Pharmalicensing

Pharmalicensing (www.pharmalicensing.com) is the premier biopharmaceutical Open Innovation resource designed for professionals involved with partnering, licensing and business development worldwide.
Actively supporting all forms of partnering and in- and out-licensing activities, Pharmalicensing utilizes the unique online Showcase Profiling & Discover services, as well as its renowned Partnering Search service leveraging its global network of industry experts, to enable companies to identify and engage with appropriate partners around the world.
Pharmalicensing is actively utilized by many tens of thousands of industry professionals each month. Pharmalicensing is a division of Cognis Group, Inc

American Pharmaceutical Review

Start receiving your free copy today. American Pharmaceutical Review is a review of the drug pipeline from the late discovery phase through manufacturing. American Pharmaceutical Review prides itself on having the most relevant, unbiased and informative editorial in the industry. You will find that all of our editorial is highly technical and written by government agencies, consultants, academics, and large pharmaceuticals companies. American Pharmaceutical Review covers several key topics that are important to drug production:Separations and purification, Drug Delivery, Biopharmaceutical Processing, Biopharmaceutical Development, Formulation development, Manufacturing, Microbiology, Instrumentation, Spectroscopy

Labiotech.eu

Labiotech.eu is the leading digital media covering the European Biotech industry. Over 100,000 monthly visitors use it to keep an eye on the business and innovations in biotechnology. Hope you'll enjoy reading our stories!

Pharmaceutical Outsourcing

Pharmaceutical Outsourcing, the journal dedicated to pharmaceutical and biopharmaceutical contract services. With regular sections on contract manufacturing, contract research, contract packaging, formulation/development services, contract analytical testing, APIs, stability testing, clinical research and other areas, we bring the most complete coverage of trends and issues in the industry to our 15,000 readers in North America.

Drug Discovery Today

Drug Discovery Today covers the whole of the preclinical drug discovery process. The reviews are cutting edge, written by experts in their respective fields and cover all aspects of drug discovery from genomic and proteomic approaches, computational drug design, medicinal chemistry and the translation of these sciences to therapies.

SelectScience

SelectScience is an independent, online information resource for the worldwide scientific community, and the home of trusted information for laboratory scientists. Discover the latest drug discovery and development technologies, products and techniques with product reviews, videos, application notes and news articles. Become a member for free today.

SAVE TO

Holiday Inn Kensington Forum

97 Cromwell Road
London SW7 4DN
United Kingdom

Holiday Inn Kensington Forum is perfectly situated in one of London’s most luxurious and beautiful areas within South Kensington.

The hotel is just 2 minutes walk from Gloucester Road tube station for convenient travel to Hyde Park, London Eye, Tower Bridge plus more of London’s top attractions. There are also easy and direct links to some major transport hubs including Victoria, Kings Cross St Pancras, Paddington and Heathrow.

This distinctive hotel in south London has so much to offer to make all guests really feel at home. The latest Holiday Inn relaunch is not just about the new look and feel for the hotel but to offer guests more benefits during their stay including a pillow menu for extra comfort during their sleep and a curved shower rail for more spacious feel.

In addition to our 906 rooms, all business guests can take advantage of our meeting and conference facilities including High Speed Internet Access and unlimited Starbucks coffee at The Academy. Our hotel’s professional event planners are on board to help take the hard work and stress away from planning your next event.

So whether you in London on business or pleasure, make the Holiday Inn London Kensington Forum your first choice of hotel and book your accommodation for our lowest internet rate guarantee.

Cookie Policy

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WHAT IS CPD?

CPD stands for Continuing Professional Development’. It is essentially a philosophy,
which maintains that in order to be effective, learning should be organised and
structured. The most common definition is:

‘A commitment to structured skills and knowledge enhancement for Personal or Professional
competence’

CPD is a common requirement of individual membership with professional bodies and
Institutes. Increasingly, employers also expect their staff to undertake regular
CPD activities.

Undertaken over a period of time, CPD ensures that educational qualifications do
not become obsolete, and allows for best practice and professional standards to
be upheld.

CPD can be undertaken through a variety of learning activities including instructor
led training courses, seminars and conferences, e:learning modules or structured
reading.

CPD AND PROFESSIONAL INSTITUTES

There are approximately 470 institutes in the UK across all industry sectors, with
a collective membership of circa 4 million professionals, and they all expect their
members to undertake CPD.

For some institutes undertaking CPD is mandatory e.g. accountancy and law, and linked
to a licence to practice, for others it’s obligatory. By ensuring that their members
undertake CPD, the professional bodies seek to ensure that professional standards,
legislative awareness and ethical practices are maintained.

CPD Schemes often run over the period of a year and the institutes generally provide
online tools for their members to record and reflect on their CPD activities.

TYPICAL CPD SCHEMES AND RECORDING OF CPD (CPD points and hours)

Professional bodies and Institutes CPD schemes are either structured as ‘Input’
or ‘Output’ based.

‘Input’ based schemes list a precise number of CPD hours that individuals must achieve
within a given time period. These schemes can also use different ‘currencies’ such
as points, merits, units or credits, where an individual must accumulate the number
required. These currencies are usually based on time i.e. 1 CPD point = 1 hour of
learning.

‘Output’ based schemes are learner centred. They require individuals to set learning
goals that align to professional competencies, or personal development objectives.
These schemes also list different ways to achieve the learning goals e.g. training
courses, seminars or e:learning, which enables an individual to complete their CPD
through their preferred mode of learning.

As a formal provider of CPD certified activities, SMI Group can provide an indication
of the learning benefit gained and the typical completion. However, it is ultimately
the responsibility of the delegate to evaluate their learning, and record it correctly
in line with their professional body’s or employers requirements.

GLOBAL CPD

Increasingly, international and emerging markets are ‘professionalising’ their workforces
and looking to the UK to benchmark educational standards. The undertaking of CPD
is now increasingly expected of any individual employed within today’s global marketplace.

CPD Certificates

We can provide a certificate for all our accredited events. To request a CPD certificate for a conference , workshop, master classes you have attended please email events@smi-online.co.uk